Avenue Therapeutics, Inc. · OTC:ATXI
Avenue Therapeutics — Investor Presentation (August 2023)
24 slides from Avenue Therapeutics's investor presentation, August 2023
Slide 1: ATXI – Corporate Presentation
ATXI – Corporate Presentation AVENUE THERAPEUTICS, INC. ⎸ NASDAQ: ATXI ⎸ AUGUST 2023
Slide 2: Forward-looking statement
Forward-looking statement This presentation contains predictive or “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of current or historical fact contained in this presentation, including statements that exp
Slide 3: Overview
Overview Our Mission: Deliver impactful therapies to patients suffering from neurologic diseases Diverse portfolio including first-in- Multiple near-term catalysts Compelling clinical and class and best-in-class assets for all three assets, with AJ201 as preclinical profiles demonstrate utilizing no
Slide 4: Diverse portfolio including first-in-class and best-in-class programs in high-value neurologic landscape
Diverse portfolio including first-in-class and best-in-class programs in high-value neurologic landscape with significant unmet patient need Pipeline Asset AJ201 BAER101 IV Tramadol Spinal and Bulbar Muscular Indication Atrophy (SBMA/Kennedy’s Epilepsy Post-operative pain Disease) Selective GABAA α2
Slide 5: Multiple potential near-term catalysts in CNS-focused pipeline
Multiple potential near-term catalysts in CNS-focused pipeline Next Indication Phase 1 Phase 2 Phase 3 Rights Milestone Spinal and Bulbar Phase 1b/2a U.S., EU, Muscular Atrophy Results Great Britain, AJ201 (SBMA) / Phase 1b/2a Expected in Canada, and Kennedy’s Disease 2024 Israel Initiate BAER101 Ep
Slide 6: AJ201 in development as novel, first-in-class treatment for SBMA
AJ201 in development as novel, first-in-class treatment for SBMA Results from Novel protein Phase 1b/2a degradation clinical trial mechanism expected of action in 2024 First mover Promising Potential advantage in preclinical “Pipeline in a disease with efficacy and Product” for no effective clinical
Slide 7: SBMA: Devastating, rare neurodegenerative disease with no FDA approved treatments
SBMA: Devastating, rare neurodegenerative disease with no FDA approved treatments for patients Brainstem (bulbar) Tongue lower motor neurons Arm muscle Spinal lower motor neurons Leg muscle • Rare, X-linked PolyQ disease primarily affecting men • Weakening of bulbar muscles affects chewing, speech a
Slide 8: Polyglutamine (PolyQ) diseases are characterized by mutant protein aggregation and progressive neurodegeneration
Polyglutamine (PolyQ) diseases are characterized by mutant protein aggregation and progressive neurodegeneration Normal Polyglutamine Expansion • 9+ neurodegenerative diseases (NDD) caused by expansion of CAG repeats encoding polyQ tracts in affected genes, resulting in aggregation of mutant protein
Slide 9: AJ201 enhances mutant AR protein degradation through unique, three-fold mechanism of action
AJ201 enhances mutant AR protein degradation and decreases neuroinflammation through unique, three-fold mechanism of action SBMA disease pathway AJ201 potential therapeutic activity Dysfunctions of Ubiquitin–proteasome system (UPS) Mutant Androgen Receptor AJ201 (AR) Degradation Nucleus Androgen Rec
Slide 10: Preclinical data demonstrate promising efficacy signals in grip test and dose-dependent mutant AR degradation
Preclinical data demonstrate promising efficacy signals in grip test and dose-dependent mutant AR degradation in SBMA models AJ201 led to improved motor function in AJ201 reduced levels and accumulation of the symptomatic animals compared with vehicle control mutant AR protein in mouse muscle tissue
Slide 11: Ongoing Phase 1b/2a study of AJ201 in SBMA patients expected to deliver final results in 2024
Ongoing Phase 1b/2a study of AJ201 in SBMA patients expected to deliver final results in 2024 Phase 1b/2a multicenter, double blind, randomized clinical trial overview Primary Objective Assessing safety, tolerability of AJ201 in subjects with clinically and genetically defined SBMA Secondary Objecti
Slide 12: BAER101 in development as potential best-in-class targeted therapy for treatment of epilepsy
BAER101 in development as potential best-in-class targeted therapy for treatment of epilepsy Safety Selective in humans in vitro profile demonstrated designed to in clinical improve trials in Existing safety Preclinical >700 patients Phase 2a trial treatments disease model in epilepsy plagued with
Slide 13: BAER-101 may address large unmet need in the epilepsy market
BAER-101 may address large unmet need in the epilepsy market Epilepsy 3–4M patients (~65M patients worldwide) U.S. • ~1M US patients are resistant to available drugs (known as drug resistant epilepsy) Prevalence • Above categories include some orphan (including pediatric) populations Chronic disease
Slide 14: BAER101 targets GABAA α2 and α3 subtypes more than α1 and α5
BAER101 targets GABAA α2 and α3 subtypes more than α1 and α5, potentially improving side effect profile compared to nonselective BZDs Predicted effect of targeting GABAA subtypes GABAA subtypes Therapeutic Effect α1 α2 α3 α5 Anti-convulsant Positive Anxiolysis Analgesia Muscle Relaxation Sedation Ne
Slide 15: BAER101 demonstrated a compelling safety profile at selected doses in 10 trials
BAER101 demonstrated a compelling safety profile at selected doses in 10 trials, as well as non-sedating tendencies Safety profile Example: subset analysis from Phase 2 generalized anxiety study • BAER101 tested in over 700 subjects (healthy volunteers and patients) Subjects experiencing sleepiness
Slide 16: BAER101 shows full suppression of seizure activity with minimal effective dose in GAERS model
BAER101 shows full suppression of seizure activity with minimal effective dose in GAERS1 model of absence epilepsy by Synapcell BAER101 Reduces SWD Incidence and Evaluation of BAER101 in GAERS Model Duration Dose-Dependently Background: • GAERS model mimics behavioral, electrophysiological and pharm
Slide 17: BAER101 is Phase 2a study ready
BAER101 is Phase 2a study ready • Opportunity to design a Phase 2a program for a strong POC signal in focal epilepsy and/or orphan indications (for example, Development and Epileptic Encephalopathies (DEEs), Lennox Gastaut Syndrome, Dravet) • For focal epilepsy: 2 Week Titration 8 Week Maintenance P
Slide 18: Tramadol has unique dual mechanism of action among IV analgesics
Tramadol has unique dual mechanism of action among IV analgesics designed to block patient’s pain signal with reduced abuse potential madol MO a A Tr Opioid Inhibitor of Norepinephrine Opioid Agonist Efficacy & Serotonin Re-uptake Blocking pain signal transmission with Less at both the spinal and br
Slide 19: Proven safety and efficacy profile demonstrated in two Phase 3 trials in over 700 patients
Proven safety and efficacy profile demonstrated in two Phase 3 trials in over 700 patients Both pain relief study models show benefit of Tramadol over placebo Study AVE-901-102 (Bunionectomy) SPID24 Study AVE-901-103 (Abdominoplasty) SPID24 IV Tramadol 50 mg achieved primary endpoint and all key sec
Slide 20: Reached Agreement with FDA on the Phase 3 Safety Study Design
Reached Agreement with FDA on the Phase 3 Safety Study Design • Met with FDA to discuss study design to address agency’s concern regarding opioid stacking • Reached agreement with the FDA on the noninferiority study design including primary endpoint and analysis approach • IV Tramadol should prove t
Slide 21: Executing to plan with multiple value-driving milestones ahead
Executing to plan with multiple value-driving milestones ahead BAER101 in AJ201 in SBMA IV Tramadol for Pain Epilepsy Compelling Phase 1 safety data in Compelling Phase 1 safety data Strong safety and efficacy profile healthy volunteers across 10 clinical trials across multiple late-stage clinical t
Slide 22: Led by experienced management team and board of directors
Led by experienced management team and board of directors Management Board of Directors Jay Kranzler MD PhD CEO, Urica Therapeutics Lindsay Rosenwald MD CEO, Fortress Biotech Alexandra MacLean David Jin Michael Ryan Neil Herskowitz MD Interim CFO VP Clinical Operations & Founder, ReGen Capital CEO P
Slide 23: Overview
Overview Our Mission: Deliver impactful therapies to patients suffering from neurologic diseases Diverse portfolio including first-in- Multiple near-term catalysts Compelling clinical and class and best-in-class assets for all three assets, with AJ201 as preclinical profiles demonstrate utilizing no
Slide 24: AVENUE.
A AVENUE. MHERAPLUTICS
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